About Event

With unprecedented levels of investment and an increasing number of programs progressing through the clinic, the establishment of robust gene therapy analytical strategies is more important than ever.

This focused, niche agenda will delve into the analytical challenges encountered in every stage of the gene therapy development life cycle, including:

  • Investigating the regulatory expectations for gene therapy analytical packages
  • Developing relevant and phase-appropriate potency assays
  • Understanding the most effective technologies for measuring empty vs full capsids
  • Learning from effective analytical comparability assays
  • Defining critical quality attributes
  • Dealing with limited amounts of material
  • Evaluating appropriate viral genome titering approaches
  • Optimizing outsourcing processes to maintain quality and safety


Companies confirmed to present their insights into these key topics include:

Bluebird Bio – Spark Therapeutics – AveXis – PTC Therapeutics – Takeda – Ultragenyx Gene Therapy – University of Florida – Voyager Therapeutics – FDA – Pfizer – Biogen – AGTC – Sanofi – Solid Biosciences – Logic Bio


Download the full event guide to find out more.